Drug Repurposing

Our initial focus was on supporting drug repurposing research using animal models of MEPAN. Repurposing projects can test thousands of FDA-approved drugs to identify existing medicines that may address MEPAN symptoms.

 

Because these drugs have already been reviewed by the FDA and tested in patients for safety, repurposing is potentially the fastest, most cost-effective path to finding a treatment. There are three repurposing projects that are underway:

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• A project led by the University of San Francisco and Perlara, with collaboration from Baylor University, Sheba Medical Center and the University of Oulu. This work has identified several drugs that may help patients, and these are now in a second stage of testing to further validate the findings. Read our blog post to learn more.

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• Pilot work at Kenyon College that is testing drug combinations in roundworms with mecr-1 deficiency which may provide clues on which drug classes could be part of a multi-modal treatment regimen for MEPAN.

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• A Perlara project to test repurposed drugs in fibroblasts from MEPAN patients. Several of these are derivative from a recent paper from Baylor University that shows iron dysregulation may underly disease pathology in MEPAN.